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Genetic Disease Articles & Analysis
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Gene therapy for glaucoma is a promising field of research focused on developing treatments to address the underlying causes of the disease, potentially offering more effective and long-lasting solutions than current options. ...
SOPHiA GENETICS (Nasdaq: SOPH), a cloud-native healthcare technology leader in data-driven medicine, today announced the next step in the partnership between the Company and Genomenon, a leading genomic intelligence company, to support better, data-driven outcomes in the rare disease and oncology fields. ...
This enhancement aims to offer more precise and comprehensive glycoprotein analysis, catering to the growing demands in the fields of biomedical research, disease biomarker discovery, and therapeutic development. Protein glycosylation, a major post-translational modification, significantly impacts protein folding, stability, and activity. Changes in glycoprotein abundance and ...
The applications of WGS are vast, impacting fields such as disease research, drug discovery, personalized medicine, food safety, agriculture, and pharmacy. ...
Recognizing the dire need for accelerated research in the field of rare diseases, the comprehensive gene therapy development solutions aim to overhaul the traditional slow and resource-intensive process of genetic research. ...
This offering provides researchers and clinicians with a powerful tool to investigate the exome, shedding light on the intricacies of the human genome and propelling our understanding of genetic diseases. In the era of precision medicine, comprehensive and precise genetic information plays a pivotal role. ...
To address this challenge, CD Genomics has developed a comprehensive Mitochondrial Diseases Panel Sequencing service that enables researchers to accurately identify mitochondrial DNA and nuclear DNA mutations and obtain a comprehensive view of the patient’s genetic makeup. ...
Application of induced pluripotent technology allows to fabricate tissues in diseased states, e.g., hereditary cardiac diseases, cardiac fibrosis, cancer and orphan diseases. ...
It takes into consideration an individual’s genetic makeup, lifestyle factors such as diet, and environmental influences when creating treatment plans to best meet their needs. ...
This collaboration could extend the reach of gene editing for liver and lung disease targets. “AskBio’s pioneering gene editing technology is a powerful complement to our modular SORT LNP genetic medicines platform, which enables the precise delivery of a wide variety of genetic cargoes to specific organs and cells, including large, ...
ByBayer AG
The company is pleased to announce the launch of the CRISPR off-target analysis service to help researchers optimize the CRISPR system, reduce off-target rates, and further explore genetic information related to human health. In recent years, CRISPR technology has significantly promoted the innovation of gene therapy for blood diseases, tumors, ...
The human genome has approximately 180,000 exomes, which are approximately 30 Mb in length, and the majority of disease-causing mutations among monogenic genetic diseases have been reported to be concentrated in the exome region. ...
Jubilant Therapeutics Inc., a clinical stage biopharmaceutical company developing precision oral medicines with enhanced therapeutic index to serve genetically defined patients suffering from cancer and autoimmune diseases, today announced that Syed Kazmi, Chief Executive Officer, will be making a business update presentation and meeting with institutional ...
WGS and Dante’s subsequent genomic reports are valuable health tools that can provide physicians and individuals with information about personalised dietary choices, predisposition to genetic diseases, genetic carrier information and more. Traditionally, DNA tests have been limited in the Baltic and Mediterranean regions due to cost and ...
” About Retinitis Pigmentosa Retinitis pigmentosa (RP), is a heterogeneous group of genetic diseases that cause retinal degeneration leading to near or complete blindness for most patients. The severe loss of photoreceptor cells that occurs in this genetic degenerative disease leads to partial or complete blindness. ...
By addressing the root cause of diseases, they are potentially capable of permanently reversing diseases with a one-time treatment. Gene editing serves as a key enabler for cell therapies when used outside the living body (ex vivo) and allows therapeutic targeting of a wide range of genetic diseases with a high unmet medical need ...
The approval is based on positive efficacy and safety results of the ILLUMINATE-C Phase 3 study of OXLUMO in patients with severe renal impairment, including those on hemodialysis. PH1 is an ultra-rare genetic disease characterized by oxalate overproduction in the liver. ...
– AMVUTTRA Demonstrated Halting or Reversal in Neuropathy Impairment with Subcutaneous Administration Once Every Three Months – – Decision Follows Positive Opinion from the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) in July 2022 – CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 20, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), ...
The 2022 RNAi Roundtable schedule is as follows: Cemdisiran, in Development for the Treatment of IgA Nephropathy (IgAN) and Other Complement-Mediated Diseases Tuesday, September 27, 10:00 am ET Alnylam Engine for Sustainable Innovation: Leadership in RNAi Platform and Human Genetics Friday, October 21, 10:00 am ET CNS Delivery and ALN-APP, in ...
– Company to Webcast Investor Event on September 30th at 8:00 p.m. ET – CAMBRIDGE, Mass.--(BUSINESS WIRE)--Sep. 13, 2022-- Alnylam Pharmaceuticals, Inc. (Nasdaq: ALNY), the leading RNAi therapeutics company, announced today that the Company will present additional data from the APOLLO-B Phase 3 study of patisiran, an investigational RNAi therapeutic in development for the treatment ...